Johnson & Johnson announced that the U.S. Food and Drug Administration (FDA) has approved INLEXZO (gemcitabine intravesical system) for the treatment of adult patients with Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors. The approval provides a new option for patients who have not responded to BCG therapy and those who are either unwilling or unable to undergo radical cystectomy, a surgery to remove the bladder.
INLEXZO is described as the first and only intravesical drug releasing system (iDRS) that offers extended local delivery of gemcitabine into the bladder. The device remains in the bladder for three weeks per treatment cycle, up to 14 cycles, and is inserted by a healthcare professional using a urinary catheter and stylet in an outpatient setting.
“When we acquired this novel therapy in 2019, our ambition was to give patients with bladder cancer a renewed sense of hope and belief,” said Jennifer Taubert, Executive Vice President, Worldwide Chairman, Innovative Medicine, Johnson & Johnson. “In an area that has seen little progress for more than 40 years, INLEXZO delivers a first-of-its-kind breakthrough innovation with a bright future ahead.”
The FDA’s decision was based on results from the SunRISe-1 Phase 2b clinical study. In this trial, 82 percent of patients treated with INLEXZO achieved a complete response—meaning no signs of cancer were found after treatment—with over half maintaining this response for at least one year.
“I see many patients that ultimately become BCG-unresponsive and often face life-altering bladder removal. These patients now may be ideal candidates for newly approved INLEXZO,” said Sia Daneshmand, M.D., principal investigator of SunRISe-1 and Professor of Urology at the Norris Comprehensive Cancer Center, Keck School of Medicine of University of Southern California. “In my experience, INLEXZO is well-tolerated and delivers clinically meaningful results. This will change the way we treat appropriate patients that haven’t responded to traditional therapy.”
Common side effects reported in the trial included urinary frequency, urinary tract infection, dysuria, micturition urgency, decreased hemoglobin levels, increased lipase levels, urinary tract pain, decreased lymphocytes count, hematuria (blood in urine), increased creatinine levels, increased potassium levels, increased AST enzyme levels, decreased sodium levels, bladder irritation and increased ALT enzyme levels.
“We are proud of the science that has brought us to this historic moment,” said John Reed, M.D., Ph.D., Executive Vice President R&D at Johnson & Johnson. “INLEXZO is a novel therapy with powerful efficacy and demonstrated safety profile. As the only major healthcare company that hosts both pharmaceuticals and medical devices, we leveraged the speed and scale of Johnson & Johnson to accelerate innovation and deliver this important therapy to patients.”
Meri-Margaret Deoudes, CEO of Bladder Cancer Advocacy Network (BCAN), commented on what this means for patient care: “At BCAN, our mission has always been to advocate for better todays and more tomorrows for everyone impacted by bladder cancer. This approval represents the kind of progress that brings new options to a community that urgently needs them,” she said. “Patients with bladder cancer need guidance and collaboration with providers to navigate bladder-sparing treatment options including newly approved treatments like INLEXZO so they can move forward feeling well-informed and confident.”
The FDA granted INLEXZO Breakthrough Therapy Designation (BTD), Real-Time Oncology Review (RTOR), and Priority Review status before its approval.
According to Johnson & Johnson’s statement about NMIBC as cited by sources such as https://www.cancer.org/cancer/types/bladder-cancer/about/key-statistics.html , non-muscle invasive bladder cancer can be classified by risk level depending on tumor size or presence of CIS lesions; approximately 10 percent of NMIBC cases involve CIS specifically. Standard care typically involves BCG immunotherapy but some patients do not respond; those individuals may face radical cystectomy—a surgery associated with significant risks especially among older adults who make up most NMIBC cases.
Johnson & Johnson says it will offer patient support through its J&J withMe program once doctor-patient decisions about using INLEXZO are made.
The company notes several contraindications including use in patients with perforated bladders or hypersensitivity reactions to gemcitabine components; it also highlights warnings related to metastatic progression if cystectomy is delayed when persistent CIS remains present.
Further details about safety information are available in prescribing documentation provided by Johnson & Johnson.